Non-randomised trial of a lipid lowering drug and a steroid for the treatment of acute myeloblastic leukaemia

ISRCTN	ISRCTN50635541
DOI	https://doi.org/10.1186/ISRCTN50635541
Protocol serial number	N/A
Sponsor	University Hospital Birmingham NHS Foundation Trust (UK)
Funder	The University Hospital Birmingham NHS Foundation Trust (UK) - paying incidental costs

Submission date: 03/07/2008
Registration date: 25/07/2008
Last edited: 28/09/2011

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Cancer

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr Mark Drayson
Scientific

Clinical Immunology and Division of Immunity and Infection
The Medical School
University of Birmingham
Edgbaston
Birmingham
B15 2TT
United Kingdom

Study information

Primary study design	Interventional
Study design	Interventional multicentre non-randomised phase II study
Secondary study design	Non randomised controlled trial
Scientific title	The use of Bezfibrate and medroxyProgesterone acetate in Acute Myeloid Leukaemia and refractory anaemia with excess of blasts (RAEB) type 2: a phase II non-randomised trial
Study acronym	BaP in AML
Study objectives	That patients with acute myeloblastic leukaemia (AML) who would not otherwise receive anti-leukaemia therapy will respond to therapy with bezafibrate and medroxyprogesterone acetate.
Ethics approval(s)	Ethics approval received from: 1. The South Birmingham Research Ethics Committee on the 9th April 2003 (ref: 5355) 2. University Hospitals Coventry and Warwickshire Research and Development Department on the 7th July 2004 (ref: NJ02/0304/EU) 3. The Research Ethics Committee of Glasgow Royal Infirmary on the 24th July 2003 (ref: 03HA010)
Health condition(s) or problem(s) studied	Elderly and relapsed high risk acute myeloid leukaemia
Intervention	1. Bezafibrate (Bezalip-Mono) 400 mg daily 2. Medroxyprogesterone acetate (Provera) 200 mg twice daily Patients will also be given a prophylactic vitamin supplement so that they are not deficient in vitamins A and D, multivitamin tablet containing minimum vitamin A 4000 units and vitamin D 400 units. Treatment was for 18 weeks. All patients were followed up to death (range of follow up was 8 days to 102 weeks from trial entry); one patient is still alive.
Intervention type	Drug
Phase	Phase II
Drug / device / biological / vaccine name(s)	Bezafibrate, medroxyprogesterone acetate
Primary outcome measure(s)	Tumour response as measured by: 1. Full blood count 2. Transfusion dependency (frequency of red blood cells/platelet transfusions) 3. Percentage blasts in bone marrow and peripheral blood pre and post BaP therapy 4. Bone marrow morphology as determined by blood smears Response will be assessed using Southwest Oncology Group (SWOG) criteria as modified from National Cancer Institute (NCI-) sponsored workshop guidelines. 1. Complete response (CR): less than 5% blasts in a marrow of sufficient cellularity with a peripheral neutrophil count greater than 1 x 10^9/l and platelet count of greater than 100 x 10^9/l determined by two evaluations not less than 4 weeks apart 2. Partial response (PR): as determined by two evaluations not less than 4 weeks apart: 2.1. In RAEB type 2 bone marrow should show greater than 50% decrease in myeloblasts, but not necessarily disappearance of marrow dyspoiesis. In peripheral blood, greater than 50% reduction in deficit from minimum normal levels (UHB haematology reference range) of the haemoglobin, neutrophil and platelet counts (if abnormal at baseline) with an absence of myeloblasts in the peripheral blood. 2.2. In AML bone marrow should show less than 15% myeloblasts with a decrease but not necessarily a disappearance of marrow dyspoiesis with an absence of Auer rods. Plus in peripheral blood there should be a greater than 50% reduction in deficit from minimum normal levels (UHB haematology reference range) of haemoglobin, neutrophil and platelet counts (if abnormal at baseline) with absence of myeloblasts in the peripheral blood. 3. Minor response (MR): decrease in frequency of infections or bleeding episodes and a 50% decrease in transfusion requirements, decrease of marrow dyspoiesis and improvement in peripheral counts but not enough to qualify for PR or CR nor progressive disease can be established 4. No change: neither the criteria for CR, PR, MR nor progressive disease can be established 5. Progressive disease: evidence of increased blasts in bone marrow or peripheral blood
Key secondary outcome measure(s)	No secondary outcome measures
Completion date	01/04/2006

Eligibility

Participant type(s)	Patient
Age group	Adult
Sex	All
Target sample size at registration	20
Key inclusion criteria	1. Patient has acute myeloid leukaemia (this can be any type of de novo or secondary AML, except acute promyelocytic leukaemia), or 2. Patient has refractory anaemia with an excess of blasts (greater than 10%) RAEB type 2 World Health Organization (WHO) criteria 3. Adult patients, either sex
Key exclusion criteria	1. Patient has acute promyelocytic leukaemia 2. Intensive chemotherapy is considered a suitable option 3. Low dose cytotoxic chemotherapy is likely to be required to control a rising blast cell count in the next month 4. Patient has a concurrent active malignancy 5. Patient has uncontrolled systemic disease (e.g. hypertension, diabetes) or severe cardiovascular disease 6. Patient is pregnant or lactating, or are potentially fertile (both males and females) and have not agreed to take adequate contraceptive precautions during the trial 7. Patient aged under 18 years
Date of first enrolment	01/06/2003
Date of final enrolment	01/04/2006

Locations

Countries of recruitment

United Kingdom
England

Study participating centre

Clinical Immunology and Division of Immunity and Infection,

Birmingham
B15 2TT
United Kingdom

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	01/04/2010		Yes	No